EP4037695A4 - Compositions oligonucléotidiques et leurs procédés d'utilisation - Google Patents
Compositions oligonucléotidiques et leurs procédés d'utilisation Download PDFInfo
- Publication number
- EP4037695A4 EP4037695A4 EP20875598.3A EP20875598A EP4037695A4 EP 4037695 A4 EP4037695 A4 EP 4037695A4 EP 20875598 A EP20875598 A EP 20875598A EP 4037695 A4 EP4037695 A4 EP 4037695A4
- Authority
- EP
- European Patent Office
- Prior art keywords
- methods
- oligonucleotide compositions
- oligonucleotide
- compositions
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
- 108091034117 Oligonucleotide Proteins 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/314—Phosphoramidates
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3521—Methyl
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Neurology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Neurosurgery (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Hospice & Palliative Care (AREA)
- Epidemiology (AREA)
- Psychiatry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962911340P | 2019-10-06 | 2019-10-06 | |
| US202062983736P | 2020-03-01 | 2020-03-01 | |
| PCT/US2020/032244 WO2020227691A2 (fr) | 2019-05-09 | 2020-05-08 | Compositions oligonucléotidiques et leurs procédés d'utilisation |
| US202063069704P | 2020-08-24 | 2020-08-24 | |
| PCT/US2020/054307 WO2021071788A2 (fr) | 2019-10-06 | 2020-10-05 | Compositions oligonucléotidiques et leurs procédés d'utilisation |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| EP4037695A2 EP4037695A2 (fr) | 2022-08-10 |
| EP4037695A4 true EP4037695A4 (fr) | 2024-03-27 |
Family
ID=75438309
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| EP20875598.3A Pending EP4037695A4 (fr) | 2019-10-06 | 2020-10-05 | Compositions oligonucléotidiques et leurs procédés d'utilisation |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20240175016A1 (fr) |
| EP (1) | EP4037695A4 (fr) |
| JP (1) | JP2022550915A (fr) |
| KR (1) | KR20220070324A (fr) |
| CN (1) | CN114585370A (fr) |
| AU (1) | AU2020363344A1 (fr) |
| BR (1) | BR112022006207A2 (fr) |
| CA (1) | CA3156176A1 (fr) |
| IL (1) | IL291974A (fr) |
| MX (1) | MX2022004102A (fr) |
| TW (1) | TW202126810A (fr) |
| WO (1) | WO2021071788A2 (fr) |
Families Citing this family (24)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2013288048A1 (en) | 2012-07-13 | 2015-01-22 | Wave Life Sciences Ltd. | Asymmetric auxiliary group |
| SG11201500232UA (en) | 2012-07-13 | 2015-04-29 | Wave Life Sciences Pte Ltd | Chiral control |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| EP3430019A4 (fr) | 2016-03-13 | 2019-10-30 | Wave Life Sciences Ltd. | Compositions et procédés de synthèse de phosphoramidite et d'oligonucléotides |
| WO2017220751A1 (fr) | 2016-06-22 | 2017-12-28 | Proqr Therapeutics Ii B.V. | Oligonucléotides d'édition d'arn monocaténaire |
| US11873316B2 (en) | 2016-11-23 | 2024-01-16 | Wave Life Sciences Ltd. | Compositions and methods for phosphoramidite and oligonucleotide synthesis |
| EP3630789A4 (fr) | 2017-06-02 | 2021-06-16 | Wave Life Sciences Ltd. | Compositions d'oligonucléotides et leurs procédés d'utilisation |
| US11603532B2 (en) | 2017-06-02 | 2023-03-14 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| CN111051281A (zh) | 2017-06-21 | 2020-04-21 | 波涛生命科学有限公司 | 用于合成的化合物、组合物和方法 |
| US20200362337A1 (en) | 2017-08-08 | 2020-11-19 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| AU2018333065B2 (en) | 2017-09-18 | 2024-04-18 | Wave Life Sciences Ltd. | Technologies for oligonucleotide preparation |
| US11596646B2 (en) | 2017-10-12 | 2023-03-07 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| WO2023152371A1 (fr) | 2022-02-14 | 2023-08-17 | Proqr Therapeutics Ii B.V. | Oligonucléotides guides pour l'édition d'acides nucléiques dans le traitement de l'hypercholestérolémie |
| WO2024013361A1 (fr) | 2022-07-15 | 2024-01-18 | Proqr Therapeutics Ii B.V. | Oligonucléotides pour édition d'arn médiée par adar et leur utilisation |
| WO2024013360A1 (fr) | 2022-07-15 | 2024-01-18 | Proqr Therapeutics Ii B.V. | Oligonucléotides chimiquement modifiés pour édition d'arn médiée par adar |
| GB202215614D0 (en) | 2022-10-21 | 2022-12-07 | Proqr Therapeutics Ii Bv | Heteroduplex rna editing oligonucleotide complexes |
| WO2024110565A1 (fr) | 2022-11-24 | 2024-05-30 | Proqr Therapeutics Ii B.V. | Oligonucléotides antisens pour le traitement de l'hémochromatose hfe héréditaire |
| GB202218090D0 (en) | 2022-12-01 | 2023-01-18 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency |
| WO2024121373A1 (fr) | 2022-12-09 | 2024-06-13 | Proqr Therapeutics Ii B.V. | Oligonucléotides antisens pour le traitement d'une maladie cardiovasculaire |
| GB202300865D0 (en) | 2023-01-20 | 2023-03-08 | Proqr Therapeutics Ii Bv | Delivery of oligonucleotides |
| WO2024175550A1 (fr) | 2023-02-20 | 2024-08-29 | Proqr Therapeutics Ii B.V. | Oligonucléotides antisens pour le traitement d'une maladie cardiovasculaire athérosclérotique |
| GB202304363D0 (en) | 2023-03-24 | 2023-05-10 | Proqr Therapeutics Ii Bv | Chemically modified antisense oligonucleotides for use in RNA editing |
| WO2024206175A1 (fr) | 2023-03-24 | 2024-10-03 | Proqr Therapeutics Ii B.V. | Oligonucléotides antisens pour le traitement de troubles neurologiques |
| WO2024200472A1 (fr) | 2023-03-27 | 2024-10-03 | Proqr Therapeutics Ii B.V. | Oligonucléotides antisens pour traitement des maladies du foie |
Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2019032612A1 (fr) * | 2017-08-08 | 2019-02-14 | Wave Life Sciences Ltd. | Compositions oligonucléotidiques et méthodes associées |
Family Cites Families (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| NZ724508A (en) * | 2014-03-18 | 2024-05-31 | Univ Of Massachusetts | Raav-based compositions and methods for treating amyotrophic lateral sclerosis |
| LT3283080T (lt) * | 2015-04-16 | 2020-05-25 | Ionis Pharmaceuticals, Inc. | C9orf72 ekspresijos moduliavimo kompozicijos |
| JP2022532169A (ja) * | 2019-05-09 | 2022-07-13 | ウェイブ ライフ サイエンシズ リミテッド | オリゴヌクレオチド組成物及びその使用方法 |
-
2020
- 2020-10-05 AU AU2020363344A patent/AU2020363344A1/en not_active Abandoned
- 2020-10-05 US US17/766,680 patent/US20240175016A1/en active Pending
- 2020-10-05 KR KR1020227015125A patent/KR20220070324A/ko unknown
- 2020-10-05 EP EP20875598.3A patent/EP4037695A4/fr active Pending
- 2020-10-05 BR BR112022006207A patent/BR112022006207A2/pt not_active Application Discontinuation
- 2020-10-05 JP JP2022520976A patent/JP2022550915A/ja active Pending
- 2020-10-05 MX MX2022004102A patent/MX2022004102A/es unknown
- 2020-10-05 IL IL291974A patent/IL291974A/en unknown
- 2020-10-05 CN CN202080069266.5A patent/CN114585370A/zh active Pending
- 2020-10-05 CA CA3156176A patent/CA3156176A1/fr active Pending
- 2020-10-05 WO PCT/US2020/054307 patent/WO2021071788A2/fr active Application Filing
- 2020-10-06 TW TW109134610A patent/TW202126810A/zh unknown
Patent Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2019032612A1 (fr) * | 2017-08-08 | 2019-02-14 | Wave Life Sciences Ltd. | Compositions oligonucléotidiques et méthodes associées |
Also Published As
| Publication number | Publication date |
|---|---|
| CA3156176A1 (fr) | 2021-04-15 |
| CN114585370A (zh) | 2022-06-03 |
| KR20220070324A (ko) | 2022-05-30 |
| WO2021071788A2 (fr) | 2021-04-15 |
| WO2021071788A3 (fr) | 2021-05-14 |
| JP2022550915A (ja) | 2022-12-05 |
| US20240175016A1 (en) | 2024-05-30 |
| MX2022004102A (es) | 2022-04-26 |
| TW202126810A (zh) | 2021-07-16 |
| AU2020363344A1 (en) | 2022-05-26 |
| IL291974A (en) | 2022-06-01 |
| EP4037695A2 (fr) | 2022-08-10 |
| BR112022006207A2 (pt) | 2022-07-26 |
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Legal Events
| Date | Code | Title | Description |
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| STAA | Information on the status of an ep patent application or granted ep patent |
Free format text: STATUS: THE INTERNATIONAL PUBLICATION HAS BEEN MADE |
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| PUAI | Public reference made under article 153(3) epc to a published international application that has entered the european phase |
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| 17P | Request for examination filed |
Effective date: 20220506 |
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| A4 | Supplementary search report drawn up and despatched |
Effective date: 20240228 |
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| RIC1 | Information provided on ipc code assigned before grant |
Ipc: A61K 48/00 20060101ALI20240222BHEP Ipc: A61K 31/7125 20060101ALI20240222BHEP Ipc: A61K 31/712 20060101ALI20240222BHEP Ipc: A61K 31/7115 20060101ALI20240222BHEP Ipc: C12N 15/113 20100101AFI20240222BHEP |